Gene therapy for children with severe bone disease
Part of Bones, joints & muscles clinical trials.
This trial tests a gene therapy that uses a child's own stem cells to treat autosomal recessive osteopetrosis (a genetic bone disease). It aims to improve bone health and reduce complications like fractures and vision loss.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be between 28 days and 2 years old and weigh at least 9 pounds.
- Have a diagnosis of autosomal recessive osteopetrosis caused by a specific gene change (TCIRG1).
- Must have adequate heart, lung, kidney, and liver function.
- Cannot have a fully matched sibling or unrelated donor available for a standard stem cell transplant.
- Must not have an active infection, cancer, or a history of seizures.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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